Company Product Description Indication Status
Adial Pharmaceuticals Inc., of Charlottesville, Va. AD-04 Genetically targeted therapy Alcohol use disorder Submitted a formal request to the FDA in support of company’s position that AD-04 should be considered eligible for an FDA expedited review program
Alexion Pharmaceuticals Inc., of Boston Ultomiris (ravulizumab) Long-acting C5 inhibitor Atypical hemolytic uremic syndrome Approved by Japan’s MHLW for use in adults and children
Blueprint Medicines Corp., of Cambridge, Mass. Ayvakyt (avapritinib) Kinase inhibitor Gastrointestinal stromal tumors European Commission granted conditional marketing authorization for use as monotherapy in adults with unresectable or metastatic disease harboring PGFRA D842V mutation
Brickell Biotech Inc., of Boulder, Colo., and Kaken Pharmaceutical Co. Ltd., Tokyo Sofpironium bromide gel, 5% Anticholinergic Primary axillary hyperhidrosis Kaken received marketing approval in Japan under brand name Ecclock
Cardiol Therapeutics Inc., of Oakville, Ontario Cardiorx Extra-strength cannabidiol formulation COVID-19 FDA cleared the IND for a phase II/III study in hospitalized patients with a prior history of, or risk factors for, cardiovascular disease
Daiichi Sankyo Co. Ltd., of Tokyo Enhertu (trastuzumab deruxtecan) HER2-directed antibody-drug conjugate Gastric cancer Approved by Japan’s MHLW for use in patients with HER2-positive unresectable advanced or recurrent disease that has progressed after chemotherapy
Gilead Sciences Inc., of Foster City, Calif., Galapagos NV, of Mechelen, Belgium, and Eisai Co. Ltd., of Tokyo Jyseleca (filgotinib) Oral JAK1 inhibitor Rheumatoid arthritis Approved by Japan’s MHLW for use in patients who have had an inadequate response to conventional therapies, including prevention of structural joint damage
Neurophth Therapeutics Inc., of Newark, Del. NR-082 (rAAV2-ND4, NFS-01) AAV ophthalmic gene therapy Leber’s hereditary optic neuropathy  FDA granted orphan designation for LHON associated with ND4 mutation
Olix Pharmaceuticals Inc., of Suwon, Republic of Korea OLX-10010 CTGF gene inhibitor Hypertrophic scars Submitted IND to FDA to test as adjunct therapy to reduce recurrence of hypertrophic scars after scar revision surgery
Oncoheroes Biosciences Inc., of Boston Volasertib PLK1 inhibitor Rhabdomyosarcoma FDA granted rare pediatric disease designation
Rakuten Medical Inc., of San Mateo, Calif. Akalux I.V. infusion 250 mg (cetuximab sarotalocan sodium) EGFR inhibitor Head and neck cancer Japan’s MHLW approved for use in patients with unresectable locally advanced or recurrent disease
Remegen Co. Ltd., of Yantai, China Disitamab vedotin (RC-48) Humanized anti-HER2 antibody-drug conjugate Urothelial cancer FDA granted breakthrough therapy designation for second-line treatment of patients with HER2-positive, locally advanced or metastatic disease who have also previously received platinum-containing chemotherapy
Spirovant Sciences Inc., of Philadelphia SPIRO-2101 Inhaled AAV gene therapy Cystic fibrosis FDA granted orphan and rare pediatric disease designations
Vertex Pharmaceuticals Inc., of Boston Kalydeco (ivacaftor) CFTR stimulator Cystic fibrosis Approved by FDA for use in children, ages 4 months to less than 6 months, who have at least 1 mutation in the CFTR gene that is responsive to Kalydeco based on clinical and/or in vitro assay data
Xiangxue Life Sciences Ltd., of Nanjing, China TAEST-16001 TCR-T program targeting tumors that are NY-ESO-1-positive Solid tumors Partner Genscript Probio said FDA cleared IND for phase I study

Notes

For more information about individual companies and/or products, see Cortellis.